Module 1 2021

30/06/2021

Some numbers (1/2)

Total vs new orphan designation conditions

2000 - 2020 •

applications for OD: 3.678 EC designation: 2.382 initial MA with OD status: 190 and 34 extension of indications

• • •

Total designations: 2.382 New conditions: 557 (23%)

The Organisation for Professionals in Regulatory Affairs

Source: https://www.ema.europa.eu/en/documents/other/orphan-medicines-figures-2000-2020_en.pdf

23

Some numbers 2/2

OD based on Significant benefit

Significant Benefit Clinically relevant advantage Major contribution to patient care

The Organisation for Professionals in Regulatory Affairs

Source: https://www.ema.europa.eu/en/documents/other/orphan-medicines-figures-2000-2020_en.pdf

24

12